It doesn't make for a very compelling news story, but the real reason why Glybera failed is because it's not that great of a drug and it loses effect over time and you can't redose with the same AAV vector. They also relied a surrogate endpoint versus actual clinical measures (as those were to confounded by the small sample size and patient variability). It just barely got approved by the EMA and based on feedback th…
The million-dollar drug (2018)
61–70 of 96 posts
Re: The million-dollar drug (2018)
#62Earlier quoted context omitted.
The shareholders are doing as much as the rest of us to help those dozens of people. It isn't fair to cast the blame on them. The real culprit here is the system of laws and controls that forbid anyone else from stepping in to fill the hole they've left. The regulations and regulators deserve to be held up as the problem here. If it wasn't illegal to compete then maybe someone would. Maybe even one of the people who…
So then why doesn't the company release the patent to the public?
A good plan should never have "and then people act against their own interests" as a step.
Re: The million-dollar drug (2018)
#63Re: The million-dollar drug (2018)
#64Earlier quoted context omitted.
So then why doesn't the company release the patent to the public?
Because it isn't in their interests. They aren't running a charity. A good plan should never have "and then people act against their own interests" as a step.
Re: The million-dollar drug (2018)
#65Earlier quoted context omitted.
The shareholders are doing as much as the rest of us to help those dozens of people. It isn't fair to cast the blame on them. The real culprit here is the system of laws and controls that forbid anyone else from stepping in to fill the hole they've left. The regulations and regulators deserve to be held up as the problem here. If it wasn't illegal to compete then maybe someone would. Maybe even one of the people who…
So then why doesn't the company release the patent to the public?
Re: The million-dollar drug (2018)
#66Earlier quoted context omitted.
A drug with high cost of production that’s not needed very often will cost a lot of money per dose. The article mentions that hundreds of millions of dollars were spent on R&D and clinical trials, and maybe a couple thousand people in the first world have this disorder. Development costs have to be recouped entirely off of first world sales, since the rest of the world isn’t going to ever pay any appreciable amount o…
How much does it cost now, after all the R&D and tests are completed? I doubt they need millions to actually produce the drug once they know how to and that it works. So they basically prefer not to produce it at all, than to sell it at affordable prices. It makes no sense to me.
Re: The million-dollar drug (2018)
#67Re: The million-dollar drug (2018)
#68Never mind non-practicing entities for software patents, but surely for patents on medicine keeping them valid when no one is making any is entirely bogus and not deserving of legal protection?
What does the patent cover in this instance? The very concept of patching the gene? Or is it the mechanism of doing so?
https://patents.google.com/patent/CA2370081C/en?q=Alipogene+....
Re: The million-dollar drug (2018)
#69Earlier quoted context omitted.
Probably not, the article mentions that the genetic mutation effects about two or three people out of a million, so that’s ~20,000 doses required to cure everyone in the world. The vast, vast majority of those people are going to be people without insurance or government provided healthcare that would ever pay enough to make a difference, so they’ve got to recoup their entire R&D expenses selling the 2,000-3,000 dose…
Means they recover non through this drug and recover all RD through other drugs. And let's not forget that this business often operates with sickeningly huge profit margins.
This is grossly misinformed
Looking at the UniQure Tax filings for 2020, They had 37M in revenue, -122M in R&D expenses, and -42M in general expenses, so they were -125M in the hole that year.
https://uniqure.gcs-web.com/static-files/8f960e6d-c40c-474e-...
Re: The million-dollar drug (2018)
#70Earlier quoted context omitted.
How much does it cost now, after all the R&D and tests are completed? I doubt they need millions to actually produce the drug once they know how to and that it works. So they basically prefer not to produce it at all, than to sell it at affordable prices. It makes no sense to me.
I think it makes no sense because you are greatly underestimating the cost to produce and market the drug, even after sunk costs. It could easily be many hundreds of thousands of per dose.
Also, I don't think marketing is a big issue for such drug which is the only available option for affected people.